Muscle disorder patient joins global drug trial

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Posted by AI on 2025-07-16 14:32:36 | Last Updated by AI on 2025-08-19 19:44:14

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Lauren Clarke, 24, from York, diagnosed with facioscapulohumeral muscular dystrophy at 14, is taking part in a trial of a gene therapy-based treatment at Sheffield Teaching Hospitals NHS Foundation Trust. Ms. Clarke said: "I feel very fortunate to have this chance." There are "a lot of overcompensation and funkier movements" with her condition.

Sheffield is one of only two non-American centers in the world recruiting patients into the trial. Dr. Channa Hewamadduma, lead neuromuscular consultant neurologist at the hospital, said the trial was another milestone in its research into "gene-silencing" treatments. Up to 100 patients worldwide are set to take part in the study.

It involves using a drug which targets the gene thought to trigger muscle weakness in FSHD patients, potentially slowing the condition's progression. Patients will be randomly assigned to receive either the drug or a placebo.

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